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CHMP opinion positive for Zokinvy as a treatment for Hutchinson-Gilford Progeria Syndrome and processing deficient progeroid laminopathies.

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Published:23rd May 2022

Eiger BioPharmaceuticals Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion recommending the European Commission approve Zokinvy, (Sarasar in the US), a first-in-class breakthrough therapy for Hutchinson-Gilford progeria syndrome (HGPS) and processing-deficient progeroid laminopathies (PL).

HGPS and PL are devastating, ultra-rare, and fatal pediatric diseases that cause dramatically accelerated aging and premature death. The main cause of death from these conditions is heart attack or stroke due to severe hardening of the arteries. Without Zokinvy treatment, children with HGPS die at an average age of 14.5 years.

The CHMP based its decision on the results of two clinical trials which showed that Zokinvy, a disease-modifying agent, lowered the risk of death in children with HGPS by 72%. Zokinvy extended life by an average of 4.3 years in children and young adults with HGPS. The science and innovation for Zokinvy spans 13 years of clinical research, including clinical trials involving almost 100 children diagnosed with progeria from 37 different countries across six continents. The U.S. approval and positive CHMP opinion are the result of a pioneering partnership between Eiger and the Progeria Research Foundation to bring Zokinvy to market and hope to patients.

Condition: Progeria
Type: drug

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